CSL312_3003 Safety and Pharmacokinetic study in Subjects 2 to 11 Years of Age with Hereditary Angioedema
- Conditions
- Hereditary angioedema (HAE)MedDRA version: 20.0Level: PTClassification code: 10019860Term: Hereditary angioedema Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-502386-13-00
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 20
Male or female, Aged 2 to 11 years, inclusive, with body weight = 10th percentile based on age, Diagnosed with clinically confirmed C1-INH HAE, Experienced = 2 HAE attacks during the 6 months before Screening
Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type III, Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks, Participation in another interventional clinical study, Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening, Currently receiving a therapy not permitted during the study, Being pregnant or breastfeeding
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluate the safety and pharmacokinetics of SC administration of CSL312 in the prophylactic treatment of pediatric subjects with C1-INH HAE;Secondary Objective: Evaluate efficacy, pharmacodynamics, and safety of CSL312 in the prophylactic treatment of pediatric subjects with C1-INH HAE;Primary end point(s): Number of subjects with treatment emergent adverse events (TEAEs), Percent of subjects with TEAEs, Number of TEAEs, TEAE rates per injection, TEAE rates per subject year, Maximum concentration (Cmax) of CSL312 at steady-state, Trough concentration (Ctrough) of CSL312 at steady-state, Time to maximum concentration (Tmax) of CSL312 at steady-state
- Secondary Outcome Measures
Name Time Method